RecruitingPHASE1INTERVENTIONAL

Vyxeos® With Clofarabine for Pediatric AML

This study is testing a new way to treat acute myeloid leukaemia (AML) in children and young adults (aged 1 to 21) where the cancer has returned or hasn't been completely cured by earlier treatments. We are looking at a combination of two medicines, Vyxeos and clofarabine. The main goal is to find the safest and most effective dose of these medicines when given together. Doctors are hoping this new combination will be better at fighting the leukaemia cells while reducing some of the long-term side effects on the heart that can be caused by older treatments. This is an early-stage study, meaning we are still learning how well it works.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Princess Maxima Center for Pediatric Oncology

Enrolment target

Start

06 Nov 2020

Estimated completion

31 Dec 2028

Relapsed Pediatric AML Refractory Pediatric AML

What is this study about?

When children and young adults are first diagnosed with a type of blood cancer called Acute Myeloid Leukaemia (AML), strong chemotherapy treatments often help about 7 out of 10 patients. However, if the leukaemia comes back, or doesn't fully respond to the first treatments, it becomes much harder to treat, and the chances of success are lower.

Doctors are always looking for better and safer ways to treat AML, especially when it returns. In the past, a common treatment combination included a medicine called daunorubicin. Unfortunately, this medicine can sometimes cause long-term heart problems, which is a big concern for young patients who have already had lots of treatment. This study is exploring a new medicine called Vyxeos, which is designed to deliver chemotherapy in a way that might be kinder to the heart.

This study combines Vyxeos with another medicine called clofarabine. Clofarabine helps to make other chemotherapy medicines work better by boosting their ability to kill cancer cells. The main purpose of this study is to find the best dose of Vyxeos and clofarabine when given together, making sure it's as effective as possible while keeping side effects manageable. This is an early-stage study, meaning it's one of the first times this specific combination is being tested in patients.

Key takeaways

This study is for children and young adults with AML that has come back or hasn't responded to treatment.
It tests a new combination of two medicines: Vyxeos and clofarabine.
The main aim is to find the best and safest dose of these medicines together.
Doctors hope this new approach could be kinder to the heart than some older treatments.
Participation involves regular hospital visits for treatment, tests, and monitoring.

Who may be eligible?

This study is looking for children and young adults aged between 1 and 21 years old who have AML. You might be suitable if your AML has come back at least a second time, or if it hasn't responded to previous standard treatments.

You could also be considered if your AML came back within a year of your first diagnosis, or if it has returned after you've had a stem cell transplant. Patients whose AML has certain 'high-risk' features in their genes might also be eligible.

There are also some important health checks to make sure you are well enough to take part. This includes having good heart, kidney, and liver function, and not having certain types of infections. You also shouldn't have Down Syndrome or a specific type of AML called acute promyelocytic leukaemia, and you can't have had any other cancer treatment in the two weeks before starting the study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 1 and 21 years old?
Has your AML come back at least for a second time, or not responded to previous treatment?
Do you have good general health, including heart, kidney, and liver function?
Have you avoided other cancer treatments in the last two weeks?
Are you able to perform daily activities with reasonable energy?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the first step is a series of checks, including a bone marrow test and a spinal fluid test, within 7 days before starting the study. These tests help doctors understand your current health and the state of your leukaemia. You will then receive the study medicines, Vyxeos and clofarabine, which are given as injections into a vein.

The study aims to find the right dose of these medicines, so some participants might receive slightly different doses initially. Throughout the study, you will have regular visits to the hospital for blood tests, check-ups, and scans to monitor your health and how the treatment is working. Doctors will also keep a close eye on any side effects. The total length of your participation will depend on how you respond to the treatment and the study plan, but you will have follow-up appointments after the main treatment period.

Potential risks and benefits

Taking part in this study could offer the chance to try a new combination therapy that might be more effective against your leukaemia, especially if standard treatments haven't worked or your leukaemia has returned. This new combination is also designed to potentially reduce some of the heart-related side effects seen with older treatments. However, like all cancer treatments, there are potential risks and side effects from the medicines, which will be carefully explained to you by the study team. These can include infections due to a weakened immune system, and other chemotherapy-related side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (13)

St. Anna Kinderspital
Verified postcode
Vienna, Austria· Recruiting
Rigshospitalet
Verified postcode
Copenhagen, Denmark· Recruiting
Universitätsklinikum Augsburg
Verified postcode
Augsburg, Germany· Recruiting
Charité Berlin
Verified postcode
Berlin, Germany· Recruiting
University Children´s Hospital III Essen
Verified postcode
Essen, Germany· Recruiting
Universitätsklinikum Frankfurt
Verified postcode
Frankfurt, Germany· Recruiting
Universitätsklinikum Hamburg-Eppendorf
Verified postcode
Hamburg, Germany· Recruiting
Clinica Pediatrica Fondazione MBBM
Verified postcode
Monza, Italy· Recruiting
Ospedale Pediatrico Bambino Gesu (OPBG)
Verified postcode
Roma, Italy· Recruiting
Princess Maxima Center
Verified postcode
Utrecht, Netherlands· Recruiting
Hospital Sant Joan de Déu
Verified postcode
Barcelona, Spain· Recruiting
Hospital Vall D'Hebron
Verified postcode
Barcelona, Spain· Recruiting

Common questions

What is AML?

AML stands for Acute Myeloid Leukaemia. It's a type of cancer that affects the blood and bone marrow, which is the soft, spongy tissue inside your bones where blood cells are made.

What does 'relapsed' or 'refractory' mean?

'Relapsed' means the leukaemia has come back after treatment. 'Refractory' means the leukaemia hasn't responded to previous treatments and is still present.

What are Vyxeos and clofarabine?

These are medicines used to treat cancer. Vyxeos is a combination of two chemotherapy drugs packaged in a special way that might help reduce side effects on the heart. Clofarabine helps other chemotherapy drugs work better against leukaemia cells.

Will this study cure my AML?

This is an early-stage study to find the best and safest dose of these medicines together. While we hope for good results, a cure cannot be guaranteed. The goal is to find better treatment options.

Can I stop participating in the study if I want to?

Yes, you can choose to leave the study at any time, for any reason. Your decision will not affect the medical care you receive in the future.

How to find out more

Loes Meijs

l.a.m.meijs-3@prinsesmaximacentrum.nl +31 6 50173349 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.